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Completed

NCT Number: NCT02801539

Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to decide if sham-RMT is a useful control condition for RMT and to choose the best ways to measure the health benefits of RMT in LOPD.

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Key information

Conditions

Glycogen Storage Disease Type II Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 18 years
  • Diagnosis of LOPD
  • On enzyme replacement therapy for ≥ 26 weeks at pretest
  • Able to follow directions for study participation
  • Able to complete a home-based RMT regimen

Exclusion criteria

  • Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic condition that would prevent meaningful study participation as determined at the discretion of the principle investigator
  • Inability to give legally effective consent
  • Inability to read and understand English

Treatment and study plan

RMT therapy using modified RMT device

Device

The exercises provided by the RMT device is intended to strengthen breathing muscles.

Sham-RMT therapy using modified RMT device

Device

The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles.

Primary outcomes

  1. Change in respiratory strength

    Time frame: baseline, 12 weeks, 6 months, 9 months

    RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).

  2. Feasibility of sham-RMT, as measured by program adherence

    Time frame: 12 weeks

    As measured by program adherence (good is considered greater than or equal to 80%). Helps determines if a future efficacy trial is warranted and if sham-RMT may be a good control condition for a future trial.

Secondary outcomes

  1. Change in maximum expiratory pressures (MEP)

    Time frame: baseline, 12 weeks, 6 months, 9 months

    Change pretest to posttest

  2. Change in 6-minute walk test (6MWT)

    Time frame: baseline, 12 weeks, 6 months, 9 months

    Change pretest to posttest

  3. Change in Gait, Stairs, Gower, and Chair (GSGC) scale

    Time frame: baseline, 12 weeks, 6 months, 9 months

    Change pretest to posttest

  4. Change in peak cough flow (PCF)

    Time frame: baseline, 12 weeks, 6 months, 9 months

    Change pretest to posttest

  5. Change in Rasch-built Pompe-specific Activity Score (R-PAct)

    Time frame: baseline, 12 weeks, 6 months, 9 months

    Change pretest to posttest

  6. Change in diaphragm thickness

    Time frame: baseline, 12 weeks, 6 months, 9 months

    change pretest to posttest

Sponsors and collaborators

Lead sponsor

Duke University

Other

Collaborators

  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Registry information

Official study title

Respiratory Muscle Training in Late-Onset Pompe Disease

Important dates

Study start
2017
Primary completion
2018
Study completion
2019
First posted
Jun 16, 2016
Registry last updated
Jul 30, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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